E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Acromegaly is a chronic metabolic disorder in which there is too much growth hormone and the body tissues gradually enlarge. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10000599 |
E.1.2 | Term | Acromegaly |
E.1.2 | System Organ Class | 10014698 - Endocrine disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
•To assess maintenance of biochemical control of CAM2029 compared to placebo |
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E.2.2 | Secondary objectives of the trial |
-to assess maintenance of biochemical control, based on GH, with CAM2029 compared to placebo; -To evaluate the safety profile of CAM2029 compared to placebo -To assess self or partner administration -To assess plasma concentrations of octreotide after administration of CAM2029 -To measure patients' satisfaction with CAM2029 and placebo -To measure the effects of CAM2029 and placebo on quality of life
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male or female patients, ≥18 years at screening - Able to provide written informed consent to participate in the trial prior to any trial related procedures are performed - Diagnosis of acromegaly by historical evidence of (persistent or recurrent) acromegaly - Treatment with a stable dose of octreotide LAR or lanreotide ATG for at least 3 months as monotherapy prior to screening - IGF-1 levels ≤1xULN at screening - Adequate liver, pancreatic, renal and bone marrow functions - Normal ECG |
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E.4 | Principal exclusion criteria |
- GH ≥2.5 μg/L at screening (cycle) • Have received medical treatment for acromegaly with pasireotide (within 6 months prior to screening), pegvisomant (within 3 months prior to screening), dopamine agonists (within 3 months prior to screening) or other investigational agents (within 30 days or 5 half-lives prior to screening [whichever is longer]) • Patients who usually take octreotide LAR or lanreotide ATG less frequently than every 4 weeks (e.g. every 6 weeks or 8 weeks) • Patients with compression of the optic chiasm causing any visual field defect for whom surgical intervention is indicated • Patients who have undergone major surgery/surgical therapy for any cause within 1 month prior screening • Patients who have undergone pituitary surgery within 6 months prior to screening • Patients who have received prior pituitary irradiation • Patients with poorly controlled diabetes mellitus (hemoglobin A1c >8.0%) |
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E.5 End points |
E.5.1 | Primary end point(s) |
• Proportion of patients with biochemical response, defined as mean IGF-1 levels ≤1x upper limit of normal (ULN) at Week 22 and Week 24 (average of the 2 measurements) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
-Proportion of patients with mean GH cycle levels <2.5 µg/L at Week 24 -Proportion of patients with mean GH cycle levels <1.0 μg/L at Week 24 - Proportion of patients/partners declared competent by healthcare professional to administer CAM2029 or placebo -Incidence of adverse events (AEs) and laboratory and electrocardiogram (ECG) abnormalities - Octreotide plasma concentrations over time - Treatment Satisfaction Questionnaire for Medication (TSQM) scores over time using all 4 domains of TSQM (effectiveness, side effects, convenience and satisfaction) - Patient satisfaction scale scores at Week 24 - Change from baseline in Acromegaly Quality of Life Questionnaire (AcroQoL) and EuroQoL 5-dimension 5-level (EQ-5D-5L) scores |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 32 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
United States |
Russian Federation |
Turkey |
Germany |
Greece |
Hungary |
Italy |
Poland |
Spain |
Sweden |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |